Compassionate Use

Earlier this month, Johnson & Johnson (J&J) announced that it will create an independent panel to review compassionate use requests and determine how the company should respond.[i] This announcement, which comes after high-profile social media campaigns and the growing popularity of the “Right to Try” state laws, may have provided a path forward to enable the dying to pursue potentially promising, experimental treatments. According to Dr. Caplan, whom J&J has appointed to lead this panel, “It used to be you would call your local news and try to beg them to cover you. Now you build this giant Twitter thing and you make the media come to you.”[i] These new platforms to pressure drug companies may finally be the catalyst to change the way they respond to patients’ request for experimental medicines. The issue of compassionate use has come a long way since the AIDS epidemic of the 1980s, when the Food and Drug Administration (FDA) set up a process, called expanded access, to help patients obtain the experimental antiretroviral drug, AZT (azidothymidine).[ii] Since its inception, the program has been a pathway for patients who have exhausted all treatment options to access investigational therapies outside of formal clinical trials. But the program has been quite controversial. In May 2006, the Abigail Alliance won an appeal suing the FDA on the grounds that patients have a constitutional right to access experimental medicines without permission from the FDA.[iii] The case was resolved when the Supreme Court declined to hear it, stating that patients do not have a right to obtain "a potentially toxic drug with no proven therapeutic benefit.”[iv] Since the ruling, there have been a number of other efforts to sidestep FDA approval in the pursuit of investigational drugs.[v],[vi] None have been more successful than the “Right to Try” measures proposed by the Goldwater Institute, which empowers states to allow terminally ill patients to access experimental therapies without FDA approval.[vii] Supporters argue that FDA involvement introduces unnecessary obstacles that overly complicate the process and discourage patients and physicians from applying. The popularity of this proposal is evidenced by the more than 20 states that have introduced “Right to Try” bills since 2014.[viii] However, critics of the bills have emphasized that drug companies, as opposed to the FDA, are the major roadblock to drug access under compassionate use.[i] The FDA cannot compel a company